National Human Genome Research Institute

National Institutes of Health U.S. Department of Health and Human Services

Young patients benefit from collaboration between NIH and Children's National Medical Center

Bethesda, Md., Tues., June 12, 2012 — A new agreement to collaborate on clinical research studies involving young children will encourage greater insight into early origin and development of disease and discovery of new treatments for rare disorders.

The Translational Research in Pediatrics Program is a joint effort from the National Institutes of Health and the NIH Clinical Center, both in Bethesda, Md., and Children's National Medical Center in Washington, D.C. The clinical expertise and infrastructure of Children's National to support a very young patient population and the state-of-the-art research facilities at the NIH Clinical Center will allow investigators to develop new studies addressing rare conditions at an earlier age.

Through the new program, NIH investigators will identify a research collaborator at Children's National, and Children's National will offer access to its Clinical Research Center for outpatient visits. This arrangement will include the delivery of general outpatient services and certain services as available, such as pulmonary function and neuropsychological testing. Children's National has authorized 20 inpatient hospital admissions under the program using private funds.

The NIH Clinical Center sees more rare disease patients than anyplace else in the nation, historically caring for those older than 2 years or who weigh more than 20 pounds. As researchers work to pinpoint the genetic causes of rare conditions and develop treatment options, they want to be able to intervene earlier in the course of diseases.

"We are grateful that Children's National is opening its doors to help us expand our research profile and establish new partnerships between our institutions that will make a difference in the lives of young children suffering from rare, and often life-threatening, disease," said NIH Clinical Center Director John I. Gallin, M.D.

Children's National Medical Center has served the nation's children for more than 140 years, and has developed expertise in the treatment of rare and genetic conditions. The hospital received an NIH Clinical and Translational Science Award in 2010, joining a consortium that aims to improve human health by transforming the research and training environment to enhance the efficiency and quality of clinical and translational research.

"This partnership is an incredible opportunity for the investigators at Children's National who work with very young patients to develop new and innovative bridges to the broad scope of research going on at NIH," says Marshall Summar, M.D., director of the Clinical Research Center at Children's National.

The National Human Genome Research Institute (NHGRI), also part of the NIH, is piloting the Translational Research in Pediatrics Program. The first collaboration teams Charles Venditti, M.D., Ph.D., investigator in NHGRI's Genetics and Molecular Biology Branch, with Kimberly Chapman, M.D., Ph.D., principal investigator, Children's Research Institute at Children's National, on a study evaluating young patients with methylmalonic acidemia. This group of inherited metabolic disorders affects between 1 in 50,000 and 80,000 babies born in the United States.

About Children's National Medical Center: Children's National Medical Center in Washington, D.C., has been serving the nation's children since 1870. Home to Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is consistently ranked among the top pediatric hospitals by U.S. News & World Report and the Leapfrog Group. Children's National is a Magnet(r) designated hospital. With 303 beds and eight regional outpatient centers, Children's National is the only exclusive provider of acute pediatric services in the Washington metropolitan area. For more information, visit , or follow us on Facebook at www.facebook.com/childrens.national and Twitter at www.twitter.com/childrenshealth.

The NIH Clinical Center (CC) is the clinical research hospital for the National Institutes of Health. Through clinical research, clinician-investigators translate laboratory discoveries into better treatments, therapies and interventions to improve the nation's health. For more information, visit http://clinicalcenter.nih.gov

NHGRI is one of the 27 institutes and centers at the National Institutes of Health, an agency of the Department of Health and Human Services. The NHGRI Division of Intramural Research develops and implements technology to understand, diagnose and treat genomic and genetic diseases. Additional information about NHGRI can be found at its website, www.genome.gov

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov. NIH...Turning Discovery into Health.